Companies are beginning to tap big data to speed drug discovery

By Peter Winter | BioWorld Insight Editor

CBiotechnology is no stranger to the creation and analysis of large amounts of data, whether it is “-omics” in nature or from patients in clinical trials. However, having access to masses of datasets is only a good starting point; sooner or later actionable information will need to be drawn from those resources.

That is why so much buzz has been created by the term “big data,” which encompasses a range of initiatives being created to develop new methods, tools and analytics, to take advantage of the very large, complex and diverse datasets being created from biomedical research.


Branford, Conn.,-based Bioxcel Corp. is working with industry partners to help improve upon its decision-making in drug discovery. The firm has developed a cloud-based big-data analytics platform, known as PharmGPS a disease area-focused, analytics engine encompassing all major and specialty therapeutic areas.

The platform draws together data and knowledge from the clinical and patient outcomes arena as well as the molecular research environment. Using a big data approach to those multiple, and often diverse datasets, key insights can be derived for the most appropriate drug discovery strategy.

Recently, the company introduced an orphan disease suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 rare and ultra-rare diseases that have been identified.

“We continue to innovate and expand the potential of PharmGPS to address the unmet medical needs of those with rare diseases,” Vimal Mehta, company chairman told BioWorld Insight. “We are providing access to this suite through a partnership model to design and develop breakthrough treatments.”

The company noted that less than 5 percent of rare diseases currently have effective treatments. Adding to the complexity is the fact that those diseases span multiple therapeutic areas. The firm’s underlying metadata and analytics for the gamut of orphan diseases, its 3,000 associated genes and hundreds of disease pathways related to therapeutic modality, is designed to address that complexity in a highly systematic manner and help partners explore innovative ideas and uncover valuable potential therapeutic approaches.

Source : BioWorld

Centrexion Therapeutics Expands Pain Pipeline with Acquisition of Three New Analgesic Candidates from Boehringer Ingelheim


Centrexion Therapeutics, a company focused on advancing chronic pain treatment with new non-opioid and non-surgical therapies, today announced the expansion of its proprietary pipeline with the acquisition of three new analgesic candidates from Boehringer Ingelheim. The company’s proprietary pipeline consists of the first and only injectable analgesic capsaicin, known as trans-capsaicin, which is currently being evaluated in clinical trials for the treatment of pain associated with chronic conditions.

Centrexion Therapeutics was founded in November 2013 and is led by former Celgene Chairman and CEO, Sol Barer, Ph.D., who chairs the company’s Board of Directors, and Jeffrey Kindler, former Chairman and CEO of Pfizer, who serves as the company’s CEO. The company is well financed having raised $58 million to-date.

“Recognizing the tremendous need for safe, non-habit-forming and effective treatments for chronic pain, we assembled a team of industry and pain treatment leaders to form Centrexion Therapeutics,” said Mr. Kindler. “The acquisition of three of Boehringer Ingelheim’s most promising pain treatment candidates strengthens our existing proprietary pipeline of therapies based on our patented and exclusive injectable trans-capsaicin. With a diversified and rapidly advancing pipeline and an experienced management team, Centrexion is poised to redefine the pain treatment market and transform the lives of people living with chronic pain.”

Pain Therapeutics Acquisition

Centrexion Therapeutics acquired three new analgesic therapies from Boehringer Ingelheim’s research and development portfolio to deepen its efforts to bring non-opioid treatment options to patients:

  • CNTX-6970 is a novel, potent and selective cytokine CCR2 antagonist with a unique analgesic profile. This small-molecule agent has shown promising efficacy in multiple animal models of inflammatory pain and has recently demonstrated safety and binding to its target in a single ascending dose Phase 1 clinical study. Centrexion Therapeutics will take over finalization of the Phase 1 program.
  • CNTX-6016 is the first of a new generation of potent and “super-selective” cannabinoid CB2 agonists. It is 20,000 times more selective for the CB2 receptor than for the CB1 receptor and thereby able to achieve pain relief without the euphoric and other side effects seen with less selective agonists. Efficacy has been shown in multiple neuropathic pain models with no central nervous system (CNS) side effects. CNTX-6016 is IND-ready.
  • CNTX-0290, a first-in-class, potent and selective somatostatin SSTR4 agonist, is a completely novel pain target. It has demonstrated very broad efficacy in multiple animal models of chronic pain and has the potential to treat inflammatory, nociceptive, neuropathic and mixed chronic pain states. CNTX-0290 is IND-ready.

“Boehringer Ingelheim is shifting its strategic focus to key CNS symptom domains with high medical need across a range of neuropsychiatric diseases, such as Alzheimer’s disease, schizophrenia and depression. As such, we are delighted to see that promising therapies from our pain portfolio are now in the capable hands of the Centrexion Therapeutics team of experienced drug developers who are first and foremost committed to improving the lives of patients coping with chronic pain, while creating value for both companies and their shareholders,” said Klaus Mendla, Global Head of Business Development and Licensing CNS at Boehringer Ingelheim. The financial terms of the Boehringer Ingelheim acquisition were not disclosed.

These programs expand the company’s current pipeline, led by CNTX-4975, a highly potent, ultrapure, synthetic form of capsaicin (a derivative of the chili plant), called trans-capsaicin, which is the first and only patented capsaicin designed to be administered via injection directly into the site of pain. CNTX-4975 was developed in a research lab at The Johns Hopkins School of Medicine by Centrexion Therapeutics Co-founder, President and Chief Scientific Officer, James Campbell, M.D., and is currently being evaluated for the treatment of knee osteoarthritis (OA) pain in humans and pet dogs and for Morton’s neuroma pain, a rare, painful nerve disorder in the foot. CNTX-4975 works by targeting the capsaicin receptor (also known as TRPV1) to inactivate the nerve fibers transmitting pain signals to the brain – a therapeutic effect that can last for months until the nerve fiber regenerates. Through its targeted delivery process and method of action, CNTX-4975 manages pain without disrupting nerve functions in the surrounding regions.

A Unique, Translational Approach to Pain Drug Development

Centrexion Therapeutics advances clinical development through a unique, dual-pronged strategy:

  • Centrexion Therapeutics studies its therapies in companion animals with the same naturally-occurring (vs. artificially–induced) pain-causing disease conditions that are common to both humans and animals. By validating the safety and therapeutic effect of its treatments in both animals and humans, the company is able to work on parallel tracks to develop human and veterinary treatments.
  • By incorporating the power of big data analytics through a strategic collaboration with BioXcel, a pioneer in cloud-based pharma big data analytics solutions for healthcare, Centrexion Therapeutics is able to rapidly mine and link vast multidisciplinary streams of data to create well-founded scientific and commercial rationales to inform and de-risk indication selection and clinical development of its pipeline.

Founders and Management Team

In addition to Mr. Kindler, the Centrexion Therapeutics management team consists of:

  • Jim Campbell, M.D., Co-founder, President and Chief Scientific Officer
    • Former Chief Executive Officer at Arcion Therapeutics
    • Professor Emeritus of Neurosurgery at Johns Hopkins University, and former President of the American Pain Society
  • Randall Stevens, M.D., Chief Medical Officer
    • Former Corporate Vice President, Head of Inflammation and Immunology Clinical Development for Celgene Corporation
    • Board certified internist and rheumatologist with more than 25 years of experience in pharmaceutical medicine
  • Peter Hanson, DVM, Ph.D., Chief Veterinary Officer
    • Former Head of Research and Development and Medical Affairs for Abbott Animal Health
    • Board certified veterinary surgeon with 20 years of experience in pharmaceutical medicine
  • Kerrie Brady, BPharm, MS, MBA, Co-founder, Chief Business Officer
    • Former Chief Operations Officer at Arcion Therapeutics
    • Experienced, international pharmaceutical executive with over 30 years of experience within big pharma and biotech companies covering regulatory, commercial, business development and operations
  • Gregg Beloff, J.D., MBA, Chief Financial Officer
    • Founder and Managing Director with Danforth Advisors, with 20 years of experience in the life science and technology industries ranging from venture-backed start-ups to publicly traded companies

View full management team bios on our website.

“With a seasoned executive like Jeff at the helm leading a world-class management team with deep expertise in pain treatment and a proven industry track record, Centrexion Therapeutics promises to be a formidable player in the chronic pain therapeutics arena,” added Dr. Barer.

About the Burden of Chronic Pain

Chronic pain affects 100 million people in the United States and 270 million people worldwide, and currently available treatments often lack adequate efficacy and are associated with numerous side effects. The total annual incremental cost of health care due to pain ranges from $560 billion to $635 billion in the U.S., which combines the medical costs of pain care and the economic costs related to disability days, lost wages and productivity. Despite the availability of a variety of pain treatment options ranging from opioids and NSAIDs, to hyaluronic acid and surgery, millions of people continue to live with chronic, debilitating pain.

About Centrexion Therapeutics

Centrexion Therapeutics, Inc., is focused on developing and commercializing new, non-opioid and non-surgical treatments for moderate to severe chronic pain. Centrexion Therapeutics recognizes the needs of over a quarter of a billion patients living with chronic pain worldwide, and aims to develop new, safer and more effective therapies that overcome the limitations and challenges associated with current pain treatments. Founded by world-renowned leaders in drug development and well-funded by key investors, Centrexion Therapeutics is building a pain treatment powerhouse to address the substantial and growing global problem of chronic pain.

For more information about Centrexion, visit

Cancer Research May Benefit from Big Data Immuno-Oncology Platform

immunoonco news


BioXcel Corporation has announced the launch of its first-in-class PharmGPS Oncology and Immuno-Oncology Suite, which uses big data technology to help biopharmaceutical companies and other stakeholders identify and prioritize the those immunotherapy candidates in their portfolios with the best potential to improve patient outcomes.

BioXcel specializes in providing cloud-based, big data solutions for companies working to discover new therapies and better classify the performance of existing products. Its proprietary PharmGPS suite allows, according to a company press release, “the extensive analysis of tumors and the impact of constituent global drug pipelines and benchmarking, novel drug targets and therapeutic approaches, including the inhibition of immune checkpoints, on standards of care and lines of therapies … [and] is comprehensive in assessing all modalities, combinations of therapies and different stages of development.”

The PharmGPS suite currently includes over 40 tumor types, 115 validated drugs and new targets, 484 unique drugs and drug combinations, and several modalities, like cellular therapies, vaccines, immunotherapies and targeted therapies, the company reported.

“We have reached an exciting inflection point in our integrated discovery ecosystem where the application of our big data technology enables the identification of therapeutic candidates with the highest probability of success to create tangible value for our partners and potentially change the cancer treatment paradigm,” said Krishnan Nandabalan, PhD, BioXcel’s president and chief scientific officer. “This platform is designed to overcome the key challenges faced by oncologists and patients by creating potentially disruptive therapies in a rational and highly efficient manner.”

The intended goal is to help companies optimize their portfolios and pipeline assets by pinpointing and prioritizing mono- and combo therapies that have the highest potential to improve patient outcomes for specific tumor types. The interface also provides an overview of the immuno-oncologylandscape, target-drug-pathway-indication networks, and scenario assessments of potential therapeutic results.

“Our first-of-its-kind big data analytics platform is integrating and expanding upon decades of knowledge in the oncology space to drive therapeutic innovation and improve patient outcomes. We believe that PharmGPS® Oncology and Immuno-Oncology Suite has the potential to become an indispensable tool for our biopharma customers and partners, and a critical asset for BioXcel as we build our own portfolio of oncology assets,” said Luca Rastelli, PhD, vice president of Oncology R&D at BioXcel.

Takeda, BioXcel Partner to Repurpose Compounds for Rare Diseases

BioXcel said today it has joined with Takeda Pharmaceutical’s U.S.-based global development group to repurpose compounds into new treatments for rare diseases. The value of the partnership was not disclosed.

BioXcel said the expertise of its Big Data Innovation Lab will be tapped in its new partnership with Takeda Development Center Americas. The “innovation lab” is an integrated product-discovery engine designed to apply recursive mapping and big data algorithms toward drug discovery.

Lab components include the Integrated Center of Xcellence—a hub for medical, clinical, commercial, scientific, IP, and technology experts—and PharmGPS™ Orphan Disease Suite, a live analytics platform that encompasses more than 9,000 rare and ultra-rare diseases, 4,000-5,000 associated genes, 1,500 disease pathways, and distinct target-indication tiles for antibody, protein, RNA, small molecule, and gene therapy modalities.

BioXcel says PharmGPS enables commercialization of orphan drugs either via de novo drug discovery, or repurposing and reformulation of older compounds based on disease severity, gene ontology, disease pathways, proteinopathy, standard of care, emerging innovation, enabling technologies, and current drug pipeline.

Discovering novel applications for existing molecules is an increasingly attractive strategy to address patient needs, while capitalizing on previous investments and de-risking clinical development. Our Big Data Innovation Lab facilitates the repurposing of the most attractive candidates applicable to a given disease,” BioXcel CEO and Chairman, Vimal Mehta, Ph.D., said in a statement.

BioXcel provides cloud-based big data solutions for biopharmas—both in discovering new products, and benchmarking pipeline innovation and marketed products.

Rare diseases do not show up among Takeda’s core therapeutic areas (cardiovascular and metabolic, CNS, immunology and respiratory, oncology, and “general medicine”).

However, the pharma giant has had a recent success in the specialty: In December, Takeda won FDA’s Breakthrough Therapy designation for its Phase III treatment oral proteasome inhibitor ixazomib (MLN9708) in relapsed or refractory systemic light-chain (AL) amyloidosis. Nearly 3,000 cases of the rare protein misfolding disorder are diagnosed in the U.S. every year. Ixazomib is also under study for multiple myeloma and other malignancies.

Takeda is the second drug developer this year to ink a partnership with BioXcel using Big Data Innovation Lab. In January, Alexion joined BioXcel to launch a big data project intended to characterize and prioritize pertinent sets of rare diseases for potential treatments.